Talecris Biotherapeutics Receives Orphan Drug Designation For Aerosolized Alpha1-Proteinase Inhibitor To Treat Alpha1-Antitrypsin Deficiency

Main Category: Immune System / Vaccines
Article Date: 08 Feb 2010 - 3:00 PDT

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Talecris Biotherapeutics, Inc. (Nasdaq: TLCR) announced that it was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the development of an aerosol formulation of Alpha1-Proteinase Inhibitor (Human, A1PI) to treat congenital alpha1-antitrypsin (AAT) deficiency. AAT deficiency is a chronic, hereditary condition that increases the risk of certain diseases, especially emphysema, which typically emerges in the fourth decade of life. Currently, there are no approved, inhaled treatments available for the treatment of AAT deficiency.

Orphan drug designation is granted to companies to encourage the development of treatments that prevent, diagnose or treat rare, life-threatening or chronic illnesses that affect fewer than 200,000 people per year in the U.S. The designation provides incentives such as tax credits and potentially seven years of market exclusivity to companies willing to support the costly research and development programs associated with developing specialized drugs for a small population of individuals.

The initiative helps to give patients suffering from rare diseases access to the same quality of treatment as other patients. Talecris received a similar orphan drug designation for the aerosolized form of AAT from the European Commission in June of 2008.

Talecris is the manufacturer of PROLASTIN® (Alpha1-Proteinase Inhibitor [Human]) an intravenous therapy that is indicated for chronic augmentation therapy among individuals who have AAT deficiency. Individuals with AAT deficiency have low serum concentrations of the A1PI protein in their blood and lungs. Augmentation therapy is administered to raise levels of the A1PI protein.

"Talecris is committed to helping patients with rare diseases for whom few treatment options exist," said Lawrence D. Stern, chairman and CEO of Talecris. "This orphan drug designation will allow us to move forward with developing an alternative method of delivering augmentation therapy for patients who prefer an inhaled mode of administration."

An important part of Talecris' aerosol development program is the exclusive partnership between Talecris and Activaero Technologies (http://www.activaero.de/en.php), an industry leader in controlled breathing technologies for inhaled therapeutic agents. Activaero's AKITA2® APIXNEB inhalation system has demonstrated consistently high drug deposition to the central and peripheral regions of the lungs in patients with AAT deficiency, regardless of disease severity.

About PROLASTIN®

PROLASTIN (Alpha1-Proteinase Inhibitor [Human]) is derived from human plasma and is administered intravenously to raise the levels of A1PI in the blood and lungs. PROLASTIN is indicated for chronic augmentation and maintenance therapy of individuals having congenital deficiency of A1PI (alpha1-antitrypsin deficiency) with clinically demonstrable panacinar emphysema. PROLASTIN was the first plasma-derived A1PI augmentation therapy to receive approval from the U.S. Food and Drug Administration (FDA) and was approved in December 1987 and launched in February 1988.

Important Safety Information

PROLASTIN, Alpha1-Proteinase Inhibitor (Human) is indicated for chronic augmentation and maintenance therapy of individuals having congenital deficiency of alpha1- PI (alpha1-antitrypsin deficiency) with clinically demonstrable panacinar emphysema. Individuals with selective IgA deficiencies who have known antibody against IgA (anti-IgA antibody) should not receive PROLASTIN, since these patients may experience severe reactions, including anaphylaxis, to IgA which may be present. In clinical studies with PROLASTIN, reactions were observed in 1.16% of infusions, the most common events being fever, light-headedness and dizziness.

PROLASTIN is made from human plasma. Products made from human plasma may carry a risk of transmitting infectious agents, e.g., viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent. There is also the possibility that unknown infectious agents may be present in such products.

For additional information on PROLASTIN, please see full prescribing information at http://www.PROLASTIN.com.

About Alpha1-Antitrypsin Deficiency

Alpha1-antitrypsin deficiency, also known as AAT deficiency or Alpha-1, is an inherited disorder that causes significant reduction in the naturally occurring protein alpha1-proteinase inhibitor . It is most common in the Caucasian population of northern Europe and North America. AAT deficiency is also the most common cause of genetic liver disease in children, and genetic emphysema (shortness of breath) in adults. Individuals suffering from AAT deficiency often develop severe obstructive pulmonary disease (COPD) causing disability and premature death. AAT deficiency is estimated to affect 200,000 people in North America and Europe.

Source
Talecris Biotherapeutics

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